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New twist discovered in Lou Gehrig’s disease

In 1939, a star hitter for the New York Yankees was diagnosed with a rare and deadly disease. The disease, amyotrophic lateral sclerosis (ALS), attacked his nerve cells, gradually weakening his muscles. Two years after ending Lou Gehrig’s baseball career, ALS took his life.

ALS remains incurable, but new insight into its causes gives reason to hope. A research team led by St. Jude Children’s Research Hospital revealed that mutations causing ALS have an unexpected toxic effect in human nerve cells.

Investigators found that mutations in a protein called TDP-43 disrupt the delivery of critical factors to the points of contact between nerve cells and muscles. To make the discovery, the scientists developed a new way to track the factors in living human nerve cells.

The research, published in the journal Neuron, opens a novel avenue in the pursuit of treatments for ALS and related disorders such as Parkinson’s and Alzheimer’s diseases. The work was led by J. Paul Taylor, MD, PhD, of St. Jude Developmental Neurobiology.